{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,23]],"date-time":"2026-10-23T19:52:34Z","timestamp":1792785154015,"version":"4.5.0"},"reference-count":42,"publisher":"Georg Thieme Verlag KG","issue":"CP","license":[{"start":{"date-parts":[[2026,8,24]],"date-time":"2026-08-24T00:00:00Z","timestamp":1787529600000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"name":"Octapharma","id":[{"id":"https:\/\/ror.org\/002k5fe57","id-type":"ROR","asserted-by":"crossref"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["TH Open"],"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:p>Introduction Prophylaxis with factor VIII (FVIII) is the standard of care for individuals with severe hemophilia A, but it is associated with a substantial economic burden. This analysis compared the efficacy, dosing, and annual treatment costs of simoctocog alfa (Nuwiq, a recombinant FVIII [rFVIII]) with five extended half-life (EHL) rFVIII concentrate products.<\/jats:p>\n                  <jats:p>Methods Matching-adjusted indirect comparisons (MAICs) were performed to compare simoctocog alfa with efanesoctocog alfa (ALTUVIIIO) and turoctocog alfa pegol (Esperoct), and results were integrated with a previously published MAIC comparing simoctocog alfa with efmoroctocog alfa (ELOCTATE), damoctocog alfa pegol (JIVI), and rurioctocog alfa pegol (ADYNOVATE). These MAIC-adjusted populations were used to develop a cost model from a United States payer perspective.<\/jats:p>\n                  <jats:p>Results Annual total costs per person were $147,058 to $492,090 lower with simoctocog alfa compared with EHL rFVIII products, corresponding to a 21% to 46% cost reduction, primarily driven by lower drug acquisition costs. Outcomes were comparable or favorable to simoctocog alfa versus turoctocog alfa pegol, efmoroctocog alfa, damoctocog alfa pegol, and rurioctocog alfa pegol. In comparison with efanesoctocog alfa (Group A), no significant differences were observed in the proportion of individuals with zero bleeds, whereas treated total annualized bleeding rate (ABR) and treated spontaneous ABR favored efanesoctocog alfa.<\/jats:p>\n                  <jats:p>Conclusion This indirect comparison suggests that personalized prophylaxis with simoctocog alfa may offer economic advantages versus EHL rFVIII products in individuals with severe hemophilia A. Clinical outcomes were broadly comparable across comparators, although treated total and spontaneous ABRs were significantly lower with efanesoctocog alfa (Group A).<\/jats:p>","DOI":"10.1055\/a-2923-0928","type":"journal-article","created":{"date-parts":[[2026,8,24]],"date-time":"2026-08-24T22:54:43Z","timestamp":1787612083000},"source":"Crossref","is-referenced-by-count":0,"special_numbering":"S","title":["Evaluating Costs and Efficacy of rFVIII Prophylaxis Using Matching-Adjusted Indirect Comparisons in Hemophilia A"],"prefix":"10.1055","volume":"10","author":[{"given":"Craig M.","family":"Kessler","sequence":"additional","affiliation":[{"name":"Lombardi Cancer Center, Medstar Georgetown University Medical Center, District of Columbia, United States","place":["Washington"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pier M.","family":"Mannucci","sequence":"additional","affiliation":[{"name":"IRCCS Ca' Granda Maggiore Policlinico Hospital Foundation, Italy","place":["Milan"],"department":["Department of Scientific Direction"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Victor","family":"Jim\u00e9nez-Yuste","sequence":"additional","affiliation":[{"name":"Hematology and Hemotherapy Unit, La Paz University Hospital (IdiPAZ), Spain","place":["Madrid"]},{"name":"Faculty of Medicine, Universidad Autonoma de Madrid, Spain","place":["Madrid"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Laurent","family":"Frenzel","sequence":"additional","affiliation":[{"name":"Department of Haematology, H\u00f4pital Necker-Enfants malades, \u00cele-de-France, France","place":["Paris"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Benoit","family":"Guillet","sequence":"additional","affiliation":[{"name":"Haemophilia Treatment Center, University Hospital Centre Rennes, Bretagne, France","place":["Rennes"]},{"name":"Inserm, EHESP, Irset (Institut de recherche en sant\u00e9, environnement et travail - UMR_S 1085), Universit\u00e9 de Rennes, Bretagne, France","place":["Rennes"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Fernando F.","family":"Corrales-Medina","sequence":"additional","affiliation":[{"name":"Miller School of Medicine, University of Miami, Florida, United States","place":["Miami"]},{"name":"University of Miami \u2013 Hemophilia Treatment Center, Florida, United States","place":["Miami"]}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"194","published-online":{"date-parts":[[2026,8,24]]},"reference":[{"key":"ref1","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1111\/hae.14046","article-title":"WFH guidelines for the management of hemophilia, 3rd edition","volume":"26","author":"A Srivastava","year":"2020","journal-title":"Haemophilia"},{"issue":"05","key":"ref2","first-page":"e301","article-title":"Long-term outcomes from prophylactic or episodic treatment of haemophilia A: a systematic review","volume":"24","author":"J O\u2019Hara","year":"2018","journal-title":"Haemophilia"},{"issue":"07","key":"ref3","doi-asserted-by":"crossref","first-page":"S339","DOI":"10.1016\/j.jval.2022.04.273","article-title":"The economic burden of severe haemophilia A without inhibitors among adults with health insurance coverage in the United States: insights from the \u201cCost of haemophilia across the US: a Socioeconomic Survey\u201d CHESS US and CHESS US+ studies","volume":"25","author":"E F Grazzi","year":"2022","journal-title":"Value Health"},{"issue":"05","key":"ref4","doi-asserted-by":"crossref","first-page":"607","DOI":"10.1111\/hae.12438","article-title":"Considerations in individualizing prophylaxis in patients with haemophilia A","volume":"20","author":"L A Valentino","year":"2014","journal-title":"Haemophilia"},{"issue":"03","key":"ref5","doi-asserted-by":"crossref","first-page":"348","DOI":"10.1111\/hae.13438","article-title":"Defining extended half-life rFVIII-A critical review of the evidence","volume":"24","author":"J Mahlangu","year":"2018","journal-title":"Haemophilia"},{"key":"ref6","doi-asserted-by":"crossref","first-page":"101164","DOI":"10.1016\/j.blre.2023.101164","article-title":"Nothing short of a revolution: novel extended half-life factor VIII replacement products and non-replacement agents reshape the treatment landscape in hemophilia A","volume":"64","author":"H AH Abdelgawad","year":"2024","journal-title":"Blood Rev"},{"key":"ref7","doi-asserted-by":"crossref","first-page":"2040620719858471","DOI":"10.1177\/2040620719858471","article-title":"Efficacy and safety of simoctocog alfa (Nuwiq\u00ae) in patients with severe hemophilia A: a review of clinical trial data from the GENA program","volume":"10","author":"T Lissitchkov","year":"2019","journal-title":"Ther Adv Hematol"},{"issue":"02","key":"ref8","doi-asserted-by":"crossref","first-page":"225","DOI":"10.1111\/hae.12793","article-title":"Novel, human cell line-derived recombinant factor VIII (human-cl rhFVIII; 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